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An Expanded Access Program of Emicizumab in Participants With Hemophilia A With Inhibitors

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ClinicalTrials.gov Identifier: NCT03154437
Expanded Access Status : Approved for marketing
First Posted : May 16, 2017
Last Update Posted : March 29, 2018
Sponsor:
Information provided by (Responsible Party):
Genentech, Inc.

Brief Summary:
This open-label, multicenter expanded access program (EAP) is designed to provide emicizumab to eligible participants with hemophilia A with factor VIII (FVIII) inhibitors before it is commercially available in the United States for the indication of hemophilia A with FVIII inhibitors. Discontinuation may occur earlier if participant or physician decides to discontinue treatment or the sponsor discontinues emicizumab clinical development.

Condition or disease Intervention/treatment
Hemophilia A Biological: Emicizumab

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Study Type : Expanded Access
Expanded Access Type : Treatment IND/Protocol
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: An Open-Label, Multicenter, Expanded Access Program for Emicizumab in Patients With Hemophilia A With Inhibitors



Intervention Details:
  • Biological: Emicizumab
    Participants will receive emicizumab at a loading dose of 3 milligrams per kilogram (mg/kg) per week subcutaneously (SC) for 4 weeks, followed by a maintenance dose of 1.5 mg/kg per week SC thereafter. Treatment with emicizumab will continue until unacceptable toxicity, withdrawal of consent, participant or physician decision to discontinue treatment, death, the participant is able to obtain commercial drug after emicizumab becomes commercially available, or the sponsor decides to discontinue emicizumab clinical development, whichever occurs first.
    Other Names:
    • ACE910
    • CH5534262

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Criteria

Inclusion Criteria:

  • Diagnosis of congenital hemophilia A of any severity and documented history of high-titer inhibitor (that is [i.e.], greater than or equal to [>/=] 5 Bethesda Units)
  • History of treatment with episodic or prophylactic bypassing agents for at least the last 24 weeks
  • >/=6 (if on an episodic bypassing agent regimen) or >/=2 (if on a prophylactic bypassing agent regimen) bleeds within 24 weeks prior to screening
  • Currently using recombinant activated factor VII (rFVIIa) or are willing to switch to rFVIIa as primary bypassing agent for the treatment of breakthrough bleeds
  • Adequate hematologic function, defined as platelet count >/= 100,000 per microliters (mcL) and hemoglobin >/=8 grams per deciliter (g/dL) at screening
  • Adequate hepatic and renal function

Exclusion Criteria:

  • Inherited or acquired bleeding disorder other than hemophilia A
  • Ongoing (or plan to receive during the study) immune tolerance induction (ITI) therapy or prophylaxis with FVIII with the exception of participants who have received a treatment regimen of FVIII prophylaxis with concurrent bypassing agent prophylaxis
  • Treatment for thromboembolic disease within 12 months before Day 1 (with the exception of previous catheter-associated thrombosis for which antithrombotic treatment is not currently ongoing) or current signs of thromboembolic disease
  • Other conditions (example [e.g.], certain autoimmune diseases) that may increase the risk of bleeding or thrombosis
  • High risk for thrombotic microangiopathy (TMA), in the investigator's judgment
  • History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
  • Use of systemic immunomodulators (e.g., interferon or rituximab) at enrollment or planned use during the study, with the exception of antiretroviral therapy
  • Treatment with any of the following: An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration before Day 1; A non-hemophilia-related investigational drug within the last 30 days or 5 half-lives before Day 1, whichever is longer; An investigational drug concurrently
  • Any serious medical condition, treatment, or abnormality in clinical laboratory tests that, in the investigator's judgment, precludes the participant's safe participation in the study

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03154437


Locations
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United States, Colorado
University of Colorado Denver, Children's Hospital
Aurora, Colorado, United States, 80045
United States, Florida
University of Miami Miller School of Medicine
Miami, Florida, United States, 33136
United States, Illinois
Rush Medical Center
Chicago, Illinois, United States, 60612
United States, Louisiana
Tulane Medical Center; Investigational/Research Pharmacy
New Orleans, Louisiana, United States, 70112
United States, Massachusetts
Boston Childrens Hospital
Boston, Massachusetts, United States, 02115
United States, Minnesota
University of Minnesota
Minneapolis, Minnesota, United States, 55455
United States, Missouri
Children's Mercy Hosp Clinics
Kansas City, Missouri, United States, 64108
United States, New Jersey
Barnabas Health Newark Beth Israel Medical Center - Pulmonary Hypertension & Lung Transplant Program
Newark, New Jersey, United States, 07112
United States, Ohio
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
United States, Oklahoma
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, United States, 73104
United States, Texas
University of Texas Southwestern Medical Center - Children's Medical Center Dallas
Dallas, Texas, United States, 75235
University of Texas Medical School
Houston, Texas, United States, 77030
United States, Utah
University of Utah
Salt Lake City, Utah, United States, 84132
United States, Washington
Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia
Seattle, Washington, United States, 98104
Sponsors and Collaborators
Genentech, Inc.
Investigators
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Study Director: Clinical Trials Hoffmann-La Roche

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Responsible Party: Genentech, Inc.
ClinicalTrials.gov Identifier: NCT03154437     History of Changes
Other Study ID Numbers: ML39356
First Posted: May 16, 2017    Key Record Dates
Last Update Posted: March 29, 2018
Last Verified: March 2018

Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Antibodies, Bispecific
Coagulants
Immunologic Factors
Physiological Effects of Drugs